EP3773637A4 - Méthodes de traitement de la drépanocytose - Google Patents

Méthodes de traitement de la drépanocytose Download PDF

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Publication number
EP3773637A4
EP3773637A4 EP19785436.7A EP19785436A EP3773637A4 EP 3773637 A4 EP3773637 A4 EP 3773637A4 EP 19785436 A EP19785436 A EP 19785436A EP 3773637 A4 EP3773637 A4 EP 3773637A4
Authority
EP
European Patent Office
Prior art keywords
treatment methods
sickle cell
cell treatment
sickle
methods
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
EP19785436.7A
Other languages
German (de)
English (en)
Other versions
EP3773637A1 (fr
Inventor
Mark A. DEWITT
David I. MARTIN
Wendy MAGIS
Jacob E. CORN
Mark C. WALTERS
Donald B. Kohn
Zulema ROMERO GARCIA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of California
University of California Berkeley
University of California San Diego UCSD
Original Assignee
University of California
University of California Berkeley
University of California San Diego UCSD
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of California, University of California Berkeley, University of California San Diego UCSD filed Critical University of California
Publication of EP3773637A1 publication Critical patent/EP3773637A1/fr
Publication of EP3773637A4 publication Critical patent/EP3773637A4/fr
Withdrawn legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP19785436.7A 2018-04-13 2019-04-10 Méthodes de traitement de la drépanocytose Withdrawn EP3773637A4 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862657412P 2018-04-13 2018-04-13
PCT/US2019/026806 WO2019199984A1 (fr) 2018-04-13 2019-04-10 Méthodes de traitement de la drépanocytose

Publications (2)

Publication Number Publication Date
EP3773637A1 EP3773637A1 (fr) 2021-02-17
EP3773637A4 true EP3773637A4 (fr) 2021-12-15

Family

ID=68163378

Family Applications (1)

Application Number Title Priority Date Filing Date
EP19785436.7A Withdrawn EP3773637A4 (fr) 2018-04-13 2019-04-10 Méthodes de traitement de la drépanocytose

Country Status (3)

Country Link
US (1) US20210155927A1 (fr)
EP (1) EP3773637A4 (fr)
WO (1) WO2019199984A1 (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
WO2020112195A1 (fr) * 2018-11-30 2020-06-04 Yale University Compositions, technologies et procédés d'utilisation de plérixafor pour améliorer l'édition de gènes
WO2025128871A2 (fr) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Nanoparticules lipidiques comprenant des molécules d'arn codant destinées à être utilisées dans l'édition de gènes et en tant que vaccins et agents thérapeutiques
WO2025174765A1 (fr) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Nanoparticules lipidiques comprenant des molécules d'arn codant destinées à être utilisées dans l'édition génique et comme vaccins et agents thérapeutiques

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016154596A1 (fr) * 2015-03-25 2016-09-29 Editas Medicine, Inc. Procédés, compositions et constituants liés à crispr/cas
WO2017053729A1 (fr) * 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Édition du génome à médiation par une nucléase de cellules primaires et leur enrichissement

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016516672A (ja) * 2013-02-28 2016-06-09 プレジデント アンド フェローズ オブ ハーバード カレッジ 幹細胞を動員するための方法および組成物
EP3851530A1 (fr) * 2015-03-26 2021-07-21 Editas Medicine, Inc. Conversion de gènes médiée par crispr/cas
WO2017123609A1 (fr) * 2016-01-12 2017-07-20 The Regents Of The University Of California Compositions et procédés pour améliorer l'édition génomique

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016154596A1 (fr) * 2015-03-25 2016-09-29 Editas Medicine, Inc. Procédés, compositions et constituants liés à crispr/cas
WO2017053729A1 (fr) * 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Édition du génome à médiation par une nucléase de cellules primaires et leur enrichissement

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
DANIEL P. DEVER ET AL: "The changing landscape of gene editing in hematopoietic stem cells : a step towards Cas9 clinical translation", CURRENT OPINION IN HEMATOLOGY, vol. 24, no. 6, 1 November 2017 (2017-11-01), US, pages 481 - 488, XP055669388, ISSN: 1065-6251, DOI: 10.1097/MOH.0000000000000385 *
DEVER DANIEL P ET AL: "CRISPR/Cas9 [beta]-globin gene targeting in human haematopoietic stem cells", NATURE, NATURE PUBLISHING GROUP UK, LONDON, vol. 539, no. 7629, 7 November 2016 (2016-11-07), pages 384 - 389, XP037555923, ISSN: 0028-0836, [retrieved on 20161107], DOI: 10.1038/NATURE20134 *
MATTHEW C CANVER AND STUART H ORKIN: "Customizing the genome as therapy for the [beta]-hemoglobinopathies", vol. 127, no. 21, 6 April 2016 (2016-04-06), pages 2536 - 2545, XP002771817, Retrieved from the Internet <URL:http://www.bloodjournal.org/content/127/21/2536?sso-checked=true> DOI: 10.1182/BLOOD-2016-01-678128 *
ROMERO ZULEMA ET AL: "Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates", MOLECULAR THERAPY, vol. 27, no. 8, 1 August 2019 (2019-08-01), US, pages 1389 - 1406, XP055857667, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2019.05.014 *
See also references of WO2019199984A1 *

Also Published As

Publication number Publication date
EP3773637A1 (fr) 2021-02-17
US20210155927A1 (en) 2021-05-27
WO2019199984A1 (fr) 2019-10-17

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